EP 4396351 A1 20240710 - COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION
Title (en)
COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION
Title (de)
VERBINDUNGEN UND VERFAHREN ZUR REDUZIERUNG DER DMPK-EXPRESSION
Title (fr)
COMPOSÉS ET PROCÉDÉS POUR RÉDUIRE L'EXPRESSION DE DMPK
Publication
Application
Priority
- US 202163239741 P 20210901
- US 2022075768 W 20220831
Abstract (en)
[origin: WO2023034868A1] Provided are oligomeric compounds, methods, and pharmaceutical compositions for DMPK the amount or activity of DMPK RNA in a cell or animal, and in certain instances reducing the amount of DMPK protein in a cell or animal. Such oligomeric compounds, methods, and pharmaceutical compositions are useful to treat type 1 myotonic dystrophy.
IPC 8 full level
C12N 15/113 (2010.01); A61K 9/00 (2006.01); A61K 31/7125 (2006.01)
CPC (source: EP)
A61K 9/08 (2013.01); A61K 47/02 (2013.01); C12N 15/1137 (2013.01); C12Y 207/11001 (2013.01); C12N 2310/11 (2013.01); C12N 2310/3125 (2013.01); C12N 2310/315 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/3341 (2013.01); C12N 2310/341 (2013.01); C12N 2310/3515 (2013.01)
C-Set (source: EP)
Designated contracting state (EPC)
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
Designated extension state (EPC)
BA ME
Designated validation state (EPC)
KH MA MD TN
DOCDB simple family (publication)
DOCDB simple family (application)
US 2022075768 W 20220831; EP 22865773 A 20220831